Founded in 1993 by brothers Tom and David Gardner, The Motley Fool helps millions of people attain financial freedom through our website, podcasts, books, newspaper column, radio show, and premium investing services.

Why BioMarin Pharmaceutical Inc. Shares Are Jumping Higher

What's happening: BioMarin Pharmaceutical(NASDAQ:BMRN) shares increased by 10% earlier today following positive midstage trial results being released for its BMN-111, a treatment for children with achondroplasia -- the most common form of human dwarfism.

Why It's happening: BioMarin Pharmaceutical is one of the most successful developers of therapies for rare disease, and the results from this trial suggest that BMN-111 could end up being yet another important win for the company.

In this phase 2 study, 10 achondroplasia patients taking a 15 microgram per kilogram dose per day of BMN-111 showed an average increase of 50% in annualized growth velocity versus their prior six month annualized growth velocity. That's enough of an improvement, if it's maintained, for BioMarin to suggest that BMN-111 could allow the roughly 24,000 patients under 18 with this condition to resume a normalized growth rate.

BioMarin plans to sit down with regulators and create a plan to move the 15 microgram dose of BMN-111 into phase 3 trials that, if successful, could lead to an eventual FDA approval. BioMarin also intends to explore whether or not a higher dose of BMN-111 could enable "catch-up" growth in the event of delayed treatment.

Author

Todd has been helping buy side portfolio managers as an independent researcher for over a decade. In 2003, Todd founded E.B. Capital Markets, LLC, a research firm providing action oriented ideas to professional investors. Todd has provided insight to a variety of publications, including SmartMoney, Barron's, and CNN/fn.
Follow @ebcapital